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Phase 3 randomized sham-controlled study of intrathecal rugonersen in Angelman syndrome
MOH_2026-03-16_015717Phase 3Not yet recruiting
- Sponsor
- OHB Pediatrics
- Start
- Mar 2026
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of rugonersen in Angelman syndrome (AS) (3 arms), sponsored by OHB Pediatrics. Planned start Mar 2026; status: not yet recruiting.
02
Drugs
rugonersen
03
Conditions
Angelman syndrome (AS)
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